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ORPHAN DRUGS - A GLOBAL STRATEGIC BUSINESS REPORT   

Research Abstract

This report analyzes the worldwide markets for Orphan Drugs in US$ Million by the following Product Segments: Biologics, and Non-Biologics. The report provides separate comprehensive analytics for the US, Japan, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2016 through 2024. Also, a five-year historic analysis is provided for these markets. Market data and analytics are derived from primary and secondary research. Company profiles are primarily based on public domain information including company URLs. The report profiles 140 companies including many key and niche players such as -

AbbVie, Inc.
Actelion Pharmaceuticals Ltd.
Alexion Pharmaceuticals, Inc.
Amgen, Inc.
AstraZeneca Plc
Bayer AG

Click here to request a full list of companies covered in the report...

Code: MCP-7443
Price: $4950
Companies: 140
Pages: 436
Date: January 2018
Market Data Tables: 49
  Status: * New Report



TABLE OF CONTENTS


  ORPHAN DRUGS (Complete Report) Pages : 436   | $4950

   Study Reliability and Reporting Limitations.....I-1  
   Disclaimers.....I-2
Data Interpretation & Reporting Level.....I-2
  
   Product Definitions and Scope of Study.....I-3  
   A Prelude.....II-1
Table 1: List of Orphan Drug Designation Criteria in Various Countries.....II-1
1$350
   Table 2: Select Orphan Diseases and their Prevalence (includes corresponding Graph/Chart).....II-21$350
   Increasing Adoption of Orphan Drugs by Manufacturers.....II-3
The 1983 Orphan Drugs Act (ODA) Kickstarts the Industry.....II-3
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   Major Orphan Drugs Legislations Worldwide.....II-5
Orphan Drugs Market on a Rapid Growth Trajectory.....II-5
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   Table 3: Orphan Drug Designations: 2010 to 2016 (includes corresponding Graph/Chart).....II-71$350
   Select Major Organizations Dealing with Rare Diseases in the US, Canada, and
  EU.....II-8
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   Incentives for Orphan Drug Designation Products in the US, EU, and Japan.....II-91$100
   Market Challenges.....II-10
Obstacles for Conducting Orphan Drug Research.....II-10
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   Competition.....II-11 1$100
   Table 4: Global Orphan Drugs Market by Leading Brand: Percentage Market Share for 2016 (includes corresponding Graph/Chart).....II-12
Orphan Drugs Remain Strong in the Pharmaceutical Pipeline.....II-12
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   Important Orphan Drugs in Pipeline.....II-131$100
   Regulatory Incentives – A Push in the Right Direction.....II-14
Growing Unmet Medical Needs – Fulfilling the Gap.....II-14
Rising Incidence of Rare Forms of Cancer – Opportunities Galore.....II-14
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   Table 5: Growing Burden of Cancer Worldwide - Number of New Cancer Cases and Cancer-Related Deaths for the Year 2012 and 2030 (includes corresponding Graph/Chart).....II-15

Table 6: Common Cause of Cancer Deaths Worldwide - Number of Deaths due to Select Types of Cancer for 2015 (includes corresponding Graph/Chart).....II-15
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   Table 7: New Cancer Cases in the World by Affected Site: 2012 (in Thousands) (includes corresponding Graph/Chart).....II-161$350
   Select Orphan Drugs for Cancer.....II-17
Rise in Incidence of Renal Cell Carcinoma.....II-17
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   Unmet Needs in AML.....II-18
Aging Population Offers Growth Potential.....II-18
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   Table 8: Global Population Statistics for the 65+ Age Group (2016) (includes corresponding Graph/Chart).....II-191$350
   Pre-Symptomatic Screening to Expand Market Base.....II-20
Asia’s Rising Awareness and Growing Population to Drive Sales.....II-20
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   Drug Repurposing Provides Therapies for Rare Diseases.....II-21
Table 9: Major Blockbuster Pharma Drugs Having Orphan Designation (includes corresponding Graph/Chart).....II-21
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   The Trend of Old Drugs Re-Surfacing as Orphan Drugs Surges.....II-22
Interest of Major Pharmaceutical Firms Signal a Shift in Commercial Dynamics.....II-22
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   Mergers and Acquisitions Drive Market Growth.....II-231$100
   Select Mergers & Acquisitions for Orphan Drugs as of the Year 2016.....II-24
Growing Scrutiny over Skyrocketing Prices.....II-24
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   Orphan Drugs with High Prices: 2016.....II-25
Table 10: Few Orphan Drug Novel Therapies with Exorbitant Prices (includes corresponding Graph/Chart).....II-25
Medical Advancements Foster Growth.....II-25
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   Full Reimbursements Critical for Spurring Uptake of High-Priced Orphan Drugs.....II-26
Table 11: Orphan Drug Prices (includes corresponding Graph/Chart).....II-26
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   Table 12: Orphan Drugs Vs Non-Orphan Drugs Average Cost (in Dollars) per Patient per Year 2012-2016 (includes corresponding Graph/Chart).....II-27

Table 13: Orphan Drugs Vs Non-Orphan Drugs Median Cost (in Dollars) per Patient per Year 2012-2016 (includes corresponding Graph/Chart).....II-27
Regulators to Scrutinize Exorbitant Pricing of Ultra-Orphan Drugs.....II-27
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   Table 14: US FDA Approval of Ultra-Orphan Non-Cancer Drugs.....II-281$350
   Strong Market Potential for Ultra-Orphan Drugs for Amyloid Light-chain
  Amyloidosis.....II-29
Increasing Patient Engagement – The PAG Trend.....II-29
1$100
   Gene Therapy and Bioprocessing Preferred over Chronic Therapies.....II-30
Companies Developing Gene Therapies.....II-30
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   International Collaboration – The Way Forward.....II-31
CNS Therapeutics to Gain Focus in the Orphan Drugs Market.....II-31
Table 15: CNS Orphan Drug Designations by Therapeutic Area: 2016 (includes corresponding Graph/Chart).....II-31
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   Manufacturers Target Orphan Indications to Curtail Competition in Broader
  Indications.....II-32
Manufacturers Use Orphan Disease to Target Larger Therapeutic Indications.....II-32
Treating Autism – The Next Big Thing?.....II-32
Sequencing Technology – The New Research Tool for Orphan Drugs.....II-32
1$100
   Innovations in Biotechnology – A Boon for Orphan Drug Research.....II-33
Aging Gene Linked to Myeloma.....II-33
Antibody with Potential to Treat Multiple Myeloma.....II-33
1$100
   Potential Treatment for Cystic Fibrosis Patients Infected with Mycobacterium
  abscessus.....II-34
Genetic Variants to Improve Treatment of Non-small Cell Lung Cancer.....II-34
1$100
   FDA Approved Orphan Drugs (as of July 2017).....II-352$200
   FDA Approved Orphan Drugs (2016).....II-372$200
   FDA Approved Orphan Drugs (2015).....II-392$200
   FDA-Approved Orphan Drugs (2015).....II-411$100
   Select Orphan drug designation: 2017 (total 433 as of november 17, 2017).....II-422$200
   Select Orphan drug designations: 2016 (total 333).....II-442$200
   The Need for Regulation.....II-46
US – The Orphan Drug Act of 1983.....II-46
1$100
   The Amended Pharmaceutical Law 1993 (Japan).....II-47
The Australian Orphan Drugs Program – 1997.....II-47
The European Regulation on Orphan Medicinal Products – 1999.....II-47
1$100
   Orphan Drugs – A Prologue.....II-48 1$100
   Table 16: Number of Incidences Determining the Orphan Status of a Disease by Region (includes corresponding Graph/Chart).....II-49

Table 17: Definitions of Orphan Diseases in Different Countries.....II-49
Orphan Drugs Research Progress.....II-49
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   Select Orphan Disease Definitions.....II-50
Glioblastoma Multiforme.....II-50
Cystic Fibrosis.....II-50
Lyosomal Storage Disorders.....II-50
Fabry Disease.....II-50
Gaucher’s Disease.....II-50
1$100
   Pompe Disease.....II-51
Huntington’s Disease.....II-51
Lymphoma.....II-51
Multiple Myeloma.....II-51
Atypical Hemolytic Uremic Syndrome (aHUS).....II-51
Chronic Myelogenous Leukemia (CML).....II-51
Cushing’s syndrome.....II-51
1$100
   Short Bowel Syndrome (SBS).....II-52
Duchenne’s Muscular Dystrophy (DMD).....II-52
Hemophilia.....II-52
Homozygous Familial Hypercholesterolemia (hoFH).....II-52
Idiopathic Pulmonary Fibrosis (IPF).....II-52
Metastatic Melanoma.....II-52
Paroxysmal Nocturnal Hemoglobinuria (PNH).....II-52
Ph+ALL.....II-52
1$100
   Ultra-Orphan Drugs.....II-53
Table 18: Select Ultra-Orphan Diseases with Global Registered Cases but No Available Prevalence Data (includes corresponding Graph/Chart).....II-53
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   Orphan Drug Categorization.....II-54
Designation of Orphan Status.....II-54
Personalized Medicine and Orphan Drugs.....II-54
1$100
   Regulatory Differences between the US and EU.....II-551$100
   TiGenix Receives FDA Orphan Drug Designation for Cx601.....II-56
FDA Grants Orphan Drug Designation for Emerald Health Pharmaceuticals lead molecule,
  EHP-101, to Treat Systemic Scleroderma.....II-56
RedHill Biopharma Receives FDA Orphan Drug Designation for MESUPRON.....II-56
FDA Awards Orphan Drug Status to uniQure’s Potential Huntington’s Therapy.....II-56
Boston Biomedical Receives Orphan Drug Designation from FDA for DSP-7888.....II-56
Amicus Receives FDA Orphan Drug Status for ATB200/AT2221.....II-56
1$100
   Roche’s Actemra/RoActemra (tocilizumab) Receives FDA Approval to Treat CAR T
  Cell-induced Cytokine Release Syndrome.....II-57
AGTC Receives FDA Orphan Drug Designation for Gene Therapy Product Candidate.....II-57
Polyneuron’ PN-1007 Receives Orphan Drug Status from European Medicines Agency.....II-57
FDA Grants Orphan Drug Designation to Versantis VS-01.....II-57
Innovate Biopharmaceuticals Applies for FDA’s Orphan Drug Designation for
  INN-108.....II-57
FDA Grants Orphan Drug Status for Biohaven’s BHV-5000.....II-57
1$100
   Mithra Receives Orphan Drug Designation for E4.....II-58
AstraZeneca Receives Orphan Designation from EMA for inebilizumab.....II-58
Boehringer Ingelheim gets FDA Orphan Drug Status for BI 836858.....II-58
Biostage gets FDA Orphan Drug Designation for Cellspan Esophageal Implant.....II-58
AbbVie Obtains FDA Orphan Drug Designation for Risankizumab.....II-58
AbbVie gets FDA Orphan Drug Designation for Veliparib.....II-58
1$100
   TiGenix Receives Orphan Drug Designation for Cx601.....II-59
Boehringer Ingelheim gets FDA Orphan Drug Designation for nintedanib.....II-59
AstraZeneca Receives FDA Orphan Drug Designation for Selumetinib.....II-59
Amarantus gets FDA Orphan Drug Designation for Eltoprazine.....II-59
AM-Pharma Secures FDA & EMA Orphan Drug Designation.....II-59
AstraZeneca’s Tremelimumab Receives FDA Orphan Drug Designation.....II-59
1$100
   BioArbitrage chooses to focus on the Dynamic Rare Disease Sector.....II-60
Acer Therapeutics ACER-002 Receives FDA Orphan Drug Designation.....II-60
1$100
   Ergomed Plc Plans to Acquire PSR Group BV.....II-61
UDG Healthcare Acquires Cambridge BioMarketing.....II-61
Horizon Pharma Acquires River Vision Development Corp. and Teprotumumab.....II-61
Sun Pharma’s Indirect arm Taro Announces Acquisition of Thallion
  Pharmaceuticals.....II-61
Johnson & Johnson Plans to Acquire Actelion.....II-61
Escend Offers Opportunity to Acquire or License ES-3000.....II-61
1$100
   Horizon Pharma Agrees to Take Over Raptor Pharmaceutical.....II-62
Savara Acquires Serendex Pharmaceuticals.....II-62
InSite Vision Inks definitive Agreement with QLT Inc.....II-62
Roche Plans to Take over Trophos.....II-62
1$100
   AbbVie, Inc. (USA).....II-63
Actelion Pharmaceuticals Ltd. (Switzerland).....II-63
1$100
   Alexion Pharmaceuticals, Inc. (USA).....II-64
Amgen, Inc. (USA).....II-64
1$100
   AstraZeneca Plc. (UK).....II-651$100
   Bayer AG (Germany).....II-66
Biogen Inc. (USA).....II-66
BioMarin Pharmaceutical Inc. (USA).....II-66
1$100
   Boehringer Ingelheim GmbH (Germany).....II-671$100
   Bristol-Myers Squibb Company (USA).....II-68
Celgene Corp. (USA).....II-68
1$100
   CEL-SCI Corporation (USA).....II-69
Concordia International Corp. (Canada).....II-69
1$100
   Eli Lilly and Company (USA).....II-70
F. Hoffmann-La Roche Ltd. (Switzerland).....II-70
1$100
   Johnson & Johnson (USA).....II-711$100
   Merck & Co., Inc. (USA).....II-72
Merck Serono International S.A. (Germany).....II-72
Novartis AG (Switzerland).....II-72
1$100
   Pfizer, Inc. (USA).....II-73
Recordati S.p.A. (Italy).....II-73
1$100
   Orphan Europe (France).....II-74
Shire Plc. (Ireland).....II-74
Sanofi SA (France).....II-74
Genzyme Corp. (USA).....II-74
1$100
   Vertex Pharmaceuticals (USA).....II-751$100
   Table 19: World Recent Past, Current & Future Analysis for Orphan Drugs by Geographic Region - US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....II-761$350
   Table 20: World Historic Review for Orphan Drugs by Geographic Region - US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....II-771$350
   Table 21: World 14-Year Perspective for Orphan Drugs by Geographic Region - Percentage Breakdown of Value Sales for US, Japan, Europe and Rest of World Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....II-781$350
   Analysis by Product Segment.....II-79
Table 22: World Recent Past, Current & Future Analysis for Orphan Biologic Drugs by Geographic Region - US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....II-79
1$350
   Table 23: World Historic Review for Orphan Biologic Drugs by Geographic Region - US, Japan, Europe and Rest of World Markets Independently Analyzed With Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....II-801$350
   Table 24: World 14-Year Perspective for Orphan Biologic Drugs by Geographic Region - Percentage Breakdown of Value Sales for US, Japan, Europe and Rest of World Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....II-811$350
   Table 25: World Recent Past, Current & Future Analysis for Orphan Non-Biologic Drugs by Geographic Region - US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....II-821$350
   Table 26: World Historic Review for Orphan Non-Biologic Drugs by Geographic Region - US, Japan, Europe and Rest of World Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....II-831$350
   Table 27: World 14-Year Perspective for Orphan Non-Biologic Drugs by Geographic Region - Percentage Breakdown of Value Sales for US, Japan, Europe and Rest of World Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....II-841$350
   A. Market Analysis.....III-1
Current and Future Analysis.....III-1
1$75
   Table 28: Prevalence and Prices of Select Orphan Drugs in the US (includes corresponding Graph/Chart).....III-21$200
   Table 29: Leading Drugs in the US Orphan Drugs Market: Market Share of Sales in Percentage for the year 2016 (includes corresponding Graph/Chart).....III-3
Orphan Drug Act – A Milestone Legislation.....III-3
1$200
   US Leads the Orphan Drug Approval Landscape.....III-4 1$75
   Table 30: Number of Orphan Drug Approvals and Designations: 1983 to 2016 (includes corresponding Graph/Chart).....III-51$200
   Table 31: Number of FDA Approved Orphan Drugs by Type: 2013-2016 (includes corresponding Graph/Chart).....III-6
Regulatory Overview.....III-6
US Orphan Drugs Policy - A Snapshot.....III-6
1$200
   Orphan Products Grants Program.....III-7
Humanitarian Use Device (HUD) Program.....III-7
1$75
   Select CDRH Approved Devices for Rare Diseases.....III-8
Medicare Reimbursement for Orphan Drugs.....III-8
Reimbursement for Orphan Drugs Not on Single-Indication List.....III-8
1$75
   Orphan Drug Modernization Plan.....III-9
Priority Review of Orphan Drugs Becomes Popular.....III-9
1$75
   FDA Review Time Reduced for Priority Review Designation.....III-10
Table 32: Majority of Orphan Drugs Undergo Priority Review: 2015 (includes corresponding Graph/Chart).....III-10
Product Approvals.....III-10
3$200
   Strategic Corporate Developments.....III-131$75
   Select Key Players.....III-149$675
   B. Market Analytics.....III-23
Table 33: The US Recent Past, Current & Future Analysis for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....III-23
1$200
   Table 34: The US Historic Review for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....III-241$200
   Table 35: The US 14-Year Perspective for Orphan Drugs by Product Segment - Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....III-251$200
   A. Market Analysis.....III-26
Current and Future Analysis.....III-26
Regulatory Overview.....III-26
Table 36: Comparison of Japanese Orphan Drug Designation System with the US and Europe.....III-26
1$200
   New Orphan Drugs in Japan: 2015-2017.....III-27 1$75
   Table 37: Japanese Orphan Drugs Designation by Therapeutic Area: 2016 (includes corresponding Graph/Chart).....III-281$200
   B. Market Analytics.....III-29
Table 38: Japanese Recent Past, Current & Future Analysis for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....III-29
1$200
   Table 39: Japanese Historic Review for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....III-301$200
   Table 40: Japanese 14-Year Perspective for Orphan Drugs by Product Segment - Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....III-311$200
   A. Market Analysis.....III-32
Current and Future Analysis.....III-32
1$75
   Table 41: Leading Drugs in the European Orphan Drugs Market: Market Share of Sales in Percentage for the year 2016 (includes corresponding Graph/Chart).....III-331$200
   Table 42: European Orphan Medicinal Product Designations since 2008 (includes corresponding Graph/Chart).....III-341$200
   Table 43: EU Orphan Designation by Therapeutic Indications: As of 2015 (includes corresponding Graph/Chart).....III-351$200
   Orphan Drug Marketing Authorizations in Europe: As of Sept 2017.....III-361$75
   Orphan Drug Marketing Authorizations in Europe: 2016.....III-37
The European Regulation on Orphan Medicinal Products - 1999.....III-37
1$75
   EU Orphan Drug Regulation Overview.....III-38
Orphan Drugs Policies Summary in Select European Countries.....III-38
1$75
   Product Approvals.....III-391$75
   Strategic Corporate Developments.....III-40
Select Key Players.....III-40
5$375
   B. Market Analytics.....III-45
Table 44: European Recent Past, Current & Future Analysis for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....III-45
1$200
   Table 45: European Historic Review for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....III-461$200
   Table 46: European 14-Year Perspective for Orphan Drugs by Product Segment - Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....III-471$200
   A. Market Analysis.....III-48
Current and Future Analysis.....III-48
Asia’s Rising Awareness and Growing Population to Drive Sales.....III-48
1$75
   Orphan Drugs Legislation in Select Asia-Pacific Countries: 2014.....III-49
Regulatory Scenario in Select Countries.....III-49
Australia.....III-49
China.....III-49
Orphan Drugs Market Set to Grow in China.....III-49
1$75
   The Chinese Food and Drug Administration (CFDA).....III-501$75
   India.....III-51
Lack of Awareness Limits the Growth of Orphan Drug Development.....III-51
Malaysia.....III-51
Malaysia to Develop a National Framework for the Management of Rare
  Diseases.....III-51
Thailand.....III-51
1$75
   Singapore.....III-52
South Africa.....III-52
South Korea.....III-52
Taiwan.....III-52
1$75
   Strategic Corporate Development.....III-531$75
   Select Key Player.....III-541$75
   B. Market Analytics.....III-55
Table 47: Rest of World Recent Past, Current & Future Analysis for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2016 through 2024 (includes corresponding Graph/Chart).....III-55
1$200
   Table 48: Rest of World Historic Review for Orphan Drugs by Product Segment - Biologic Drugs and Non-Biologic Drugs Markets Independently Analyzed with Annual Sales Figures in US$ Million for Years 2011 through 2015 (includes corresponding Graph/Chart).....III-561$200
   Table 49: Rest of World 14-Year Perspective for Orphan Drugs by Product Segment - Percentage Breakdown of Value Sales for Biologic Drugs and Non-Biologic Drugs Markets for Years 2011, 2017 & 2024 (includes corresponding Graph/Chart).....III-571$200
  
Total Companies Profiled: 140 (including Divisions/Subsidiaries - 151)

Region/Country Players

The United States 77 Canada 8 Japan 8 Europe 51 France 10 Germany 4 The United Kingdom 7 Italy 3 Rest of Europe 27 Asia-Pacific (Excluding Japan) 3 Middle East 4
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